US FDA approves GSK treatment for myelofibrosis

GSK announced on Monday that the US Food and Drug Administration (FDA) has granted approval for ‘Ojjaara’, or momelotinib, as a treatment for adults suffering from intermediate or high-risk myelofibrosis with anaemia, including cases of primary myelofibrosis or secondary myelofibrosis.

The FTSE 100 pharmaceutical giant said Ojjaara, an oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor, is administered once daily and is the only authorised medicine that addresses key symptoms of the disease, such as anaemia, constitutional symptoms, and enlarged spleen.

It said myelofibrosis, a type of blood cancer, impacts around 25,000 patients in the US.

The disease can cause drastically low blood counts, leading to anaemia and thrombocytopaenia. Symptoms include fatigue, night sweats, and bone pain.

At the point of diagnosis, about 40% of patients show moderate to severe anaemia, and as the disease progresses, almost all patients are expected to develop anaemia, and over 30% are likely to stop their treatment because of it.

Such patients frequently require blood transfusions, and those dependent on transfusions face a reduced life expectancy.

Supporting the FDA's endorsement of momelotinib were findings from the pivotal MOMENTUM study and a subset of anaemic adult patients from the SIMPLIFY-1 phase three trial.

GSK said the MOMENTUM trial showed statistically significant results in alleviating primary symptoms of the disease when compared to danazol.

The SIMPLIFY-1 trial meanwhile compared the efficacy and safety of momelotinib to ruxolitinib, focusing on patients who hadn't previously been treated with a JAK-inhibitor therapy.

Clinical trials highlighted several common side effects, including thrombocytopaenia, haemorrhage, bacterial infections, fatigue, dizziness, diarrhoea, and nausea.

“The vast majority of myelofibrosis patients eventually develop anaemia, causing them to discontinue treatments and require transfusions,” said Nina Mojas, senior vice-president of oncology global product strategy at GSK.

“Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community.

“We look forward to helping improve outcomes in this difficult-to-treat blood cancer.”

Reporting by Josh White for Sharecast.com.